16
Jul
Azafaros press release: Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C
We are pleased to share the latest press release from Azafaros related
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25
Apr
Sanofi Press Release: Sanofi’s AMETHIST trial for GM2 gangliosidosis discontinued
Today it was announced by Sanofi that the AMETHIST phase 3 study of venglustat
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12
Mar
Azafaros RAINBOW study update: Completion of 12 week phase 2
Azafaros has just announced the successful completion of its 12-week Phase 2
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22
Jan
Patient-Focused Drug Development (PFDD) meeting with the FDA for GM2 Gangliosidosis
Our friends at the National Tay-Sachs & Allied Diseases Association have been
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28
Feb
WORLDSymposium 2023 poster – Empowering the GM1 and GM2 community with a co-created education resource about a natural history study
In February 2023 we were delighted to present a poster at the
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28
Feb
WORLDSymposium 2023 poster – Collaboration between patient advocacy and industry to create a master protocol to investigate the novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosides and Ataxia-telangiectasia
In February 2023 we were delighted to present a poster at the
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28
Feb
WORLDSymposium 2023 poster – Cherry red spot: Evidence that its identification can speed up the diagnosis of GM2 gangliosdosis
In February 2023 we were delighted to present a poster at the
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15
Mar
WORLDSymposium 2021 poster
In February 2021 we were delighted to present our latest poster alongside
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15
Mar
National Organization for Rare Disorders (NORD) Summit 2020 Poster
In October 2020 we were delighted to present alongside our registry partners
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